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Huntington's disease (HD)

Huntingtin (HTT)

Cell culture and mouse studies suggest zinc finger proteins (ZFPs) could help treat HD. In a patient-derived mesothelial cell line, a ZFP designed to bind the extended CAG repeats of HTT in patients with HD repressed HTT expression. In a mouse model of HD, brain injection of an adenoviral vector expressing the ZFP restored motor coordination to levels comparable to those in healthy controls. Next steps include testing alternative gene therapy vectors to optimize expression and running longer-term efficacy studies in additional HD models.

SciBX 5(42); doi:10.1038/scibx.2012.1119
Published online Oct. 25, 2012

Patent application filed; available for licensing

Garriga-Canut, M. et al. Proc. Natl. Acad. Sci. USA; published online
Oct. 10, 2012;
Contact: Mark Isalan, Centre for Genomic Regulation, Barcelona, Spain